By Alex Keown
The U.S. Food and Drug Administration (FDA) has done another about-face. Days after rejecting Regeneron’s attempt to secure a supplemental approval for Eylea, the FDA has now approved a new dosing label for the drug in wet age-related macular degeneration (wet AMD).
Eylea, (aflibercept), a vascular endothelial growth factor (VEGF) inhibitor, has already been approved to treat wet AMD patients as a monthly injection for the first three months of use, then once every month or two months. Regeneron had been working toward a new dosing level where the injection would only have to be administered into a patient’s eye every three months. After some discussion with the FDA, that hope is now a reality.
In wet AMD, abnormal blood vessels leak fluid into the eye, ultimately causing damage and blindness. Affecting more than 20 million people globally, wet AMD is the leading cause of severe vision loss and legal blindness in people over the age of 65.
Regeneron Chief Scientific Officer George D. Yancopoulos said the company is pleased with the FDA’s change of heart.
“Providing information to retinal physicians about the visual outcomes with a modified 12-week dosing schedule will help physicians make the most informed choices in treating patients suffering from wet age-related macular degeneration,” Yancopoulos said in a statement.
The turnaround for the FDA was spectacularly short. It was only four days ago the regulatory agency issued a Complete Response Letter for the supplemental application. At the time, the FDA said it issued the rejection over “ongoing labeling discussions.” What those label discussions were was never disclosed. Regeneron said on Monday that it anticipated completing its discussions with the FDA and receiving a final verdict within two months’ time. Those discussions clearly didn’t take long at all.
The FDA has made several notable turnarounds for drugs it had once rejected. In April Alkermes saw the FDA accept a New Drug Application for a major depressive disorder treatment one month after rejecting the NDA. In 2017 the FDA did an about-face on Amicus’ Fabry disease treatment, as well as Eli Lilly’s rheumatoid arthritis drug baricitinib and TherapeuticsMD, Inc.’s vaginal pain medicine TX-004HR.
Regeneron said the new dosing indication will now be included in the Eylea label. Shares of Regeneron climbed to a morning high of $377.47 on the news.
Regeneron’s Eylea brings in about $5 billion annually for the company and remains its key lynchpin for revenue. Eylea has been approved as a treatment for wet AMD, macular edema following retinal vein occlusion and diabetic macular edema.
With the updated dosing for Eylea approved, Regeneron now has some breathing room as it awaits an FDA decision for a potential rival wet AMD treatment from Novartis, the Swiss drug giant. Novartis’ RTH258 has not yet been approved by the FDA, but has been shown to be superior against Eylea in wet AMD in clinical trials. During a presentation at the American Academy of Ophthalmology last year, RTH258 showed superiority versus Regeneron’s aflibercept (Eylea) in key secondary endpoints reflective of disease activity in patients with wet AMD. Brolucizumab (RTH258) is a humanized single-chain antibody fragment (scFv) designed to inhibit vascular endothelial growth factor (VEGF). Patients treated with RTH258 showed fewer signs of specific disease activity than patients treated with Eylea, according to the presentation.
With the superiority shown in that data, Novartis is clearly eying taking a significant slice of the Eylea market share. The Swiss pharma company is eying a 2019 launch for RTH258.